A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®

Official title

A Multinational, Prospective, Open Labelled, Non-controlled, Non-interventional Post-authorisation Study of Turoctocog Alfa Pegol (N8-GP) During Long-term Routine Prophylaxis and Treatment of Bleeding Episodes in Patients With Haemophilia A

Overview

This study will collect information on side effects and how well Esperoct® (turoctocog alfa pegol (N8-GP)) works during long-term treatment (prophylaxis) in males with haemophilia A. Participants in this study will get the same treatment as they would normally get, if they were not participating in the study. All visits at the clinic are done in the same way as participants are used to, when visiting their doctor. During visits at the clinic participants might be asked for some relevant tests if considered useful by the study doctor. During the visits the study doctor might ask if participants had any side effects since the last study visit. Participants will be asked to note down in their own diary the number of bleeds and how these were treated, as well as their regular prophylaxis. Participation in the study will last for about 5-7 years, depending on when participants join the study. Participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.

Interventions

• Drug Turoctocog alfa pegol (N8-GP)
  Patients will be treated with commercially available N8-GP for prophylaxis and treatment of bleeding episodes according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available N8-GP has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.

Primary outcome measures

• Number of Adverse Events (AEs) reported during the study period [Time frame: From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years]

Secondary outcome measures (1)

• Number of Serious Adverse Events (SAEs) reported during the study period [Time frame: From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years]

Eligibility criteria

Inclusion criteria

• Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
• The decision to initiate treatment with commercially available Esperoct® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
• Male patients of all ages, according to local label, are allowed in this study
• Diagnosis of severe or moderate Haemophilia A

Exclusion criteria

• Previous participation in this study. Participation is defined as having given informed consent in this study
• Known or suspected hypersensitivity to N8-GP or related products
• Mental incapacity, unwillingness or language barriers precluding adequate understanding and cooperation
• Clinical suspicion or presence of FVIII inhibitors at time of inclusion

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Italy · 4 centers

• Azienda Ospedaliera-Universitaria Parma — Parma
• Policlinico Umberto I Sezione Ematologia — Roma
• A.O.U Città Salute Scienza Torino — Torino
• Ospedale San Bortolo — Vicenza

Lithuania · 4 centers

• Hospital of LUHS "Kauno Klinikos" — Kaunas
• Republican Panevezys Hospital, Public consultation clinic — Panevezys
• Vilnius University hospital Santaros klinikos — Vilnius
• Vilnius University hospital Santaros klinikos — Vilnius

Greece · 3 centers

• "Laiko" General Hospital of Athens — Athens
• "Laiko" General Hospital of Athens — Athens
• Aghia Sophia Childrens' Hospital — Athens

Czechia · 2 centers

• FN Brno odd. hematologie — Brno
• FN HK - IV. Interni hematologicka klinika — Hradec Králové

Estonia · 2 centers

• North Estonia Medical Centre Foundation — Tallinn
• Tartu University Hospital Haematology Clinic — Tartu

Germany · 2 centers

• Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain — Berlin
• Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie — Bonn

Portugal · 2 centers

• Centro Hospitalar Lisboa Norte-HSM — Lisbon
• ULS São João, E.P.E. — Porto

Austria · 1 center

• AKH - Klin. Abt. f. Haematologie u. Haemostaseologie — Vienna

Bulgaria · 1 center

• UMHAT "Tsaritsa Yoanna-ISUL" — Sofia

Croatia · 1 center

• KBC Zagreb, Rebro, Hemofilija centar — Zagreb

Hungary · 1 center

• MH Eü. Központ -Orszagos Haemophilia Kozpont — Budapest

Slovakia · 1 center

• Unilabs Slovensko, s. r. o. — Košice

Slovenia · 1 center

• PeK - University Children's Hospital, Department of haematology — Ljubljana

Spain · 1 center

• Hospital Regional Universitario de Málaga — Málaga

Switzerland · 1 center

• Kinderspital Hämatologie, Zürich — Zurich

Identifiers

NCT: NCT04574076 · NN7088-4029 · EUPAS36536 · U1111-1235-6007

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗